EASL-AASLD Delphi consensus statement on surrogate endpoints and real-world evidence in primary biliary cholangitis
International consensus guidelines now provide clear frameworks for approving promising liver disease treatments previously stuck in regulatory limbo.
A joint EASL-AASLD modified Delphi process involving 62 international panelists (clinicians, methodologists, regulators, industry, patient representatives) produced 16 consensus statements and 42 recommendations to standardize surrogate endpoint use, real-world evidence integration, and patient-reported outcomes in PBC clinical trials. This consensus directly addresses the regulatory gap blocking full approval of 3 conditionally approved second-line PBC therapies, providing a pragmatic, operationally ready framework.
What the study was
- Study design
- Modified Delphi consensus process
- Population
- Primary biliary cholangitis patients (rare autoimmune liver disease)
- Sample size
- 62
- Category
- Treatment Innovation
- Maturity
- Validated
- Journal
- Journal of Hepatology
Why it surfaced
Joint EASL-AASLD consensus for rare liver disease PBC — directly enables regulatory pathway for full approval of 3 conditionally approved therapies; 62 panelists, 88% response rate, 42 actionable recommendations; J Hepatol flagship journal; immediately applicable to ongoing PBC drug development
A plain-language summary of published research — not medical advice. Talk to a clinician about your own care.